Aegerion's Rare Disease Drug Launch Stalls

On Tuesday night, Aegerion Pharmaceuticals (AEGR) reported weaker-than-expected first quarter Juxtapid sales and reduced 2014 sales guidance. What looked like a fast and promising start to a rare disease drug launch last summer has now stalled, and management's ability to recover is being questioned.

Aegerion shares are down 29% to $31.15 in Wednesday trading.

Aegerion reported Juxtapid net sales of $27 million in the March quarter, below consensus estimate of $33 million. Management blamed the shortfall primarily on delays in orders from the Brazil. U.S. Juxtapid sales grew 24% quarter over quarter, but Aegerion admitted to a slowdown in prescriptions due to the harsh winter weather. The pace of Juxtapid prescriptions picked up in March and continued into April. Aegerion refused to disclose the number of new Juxtapid patients added in the first quarter -- a departure from the company's past practice during quarterly conference calls.

Juxtapid sales guidance for 2014 was cut back to $180-200 million from $190-210 million, with delays in Brazilian orders once again being blamed, according to Aegerion.

Given weak first quarter performance, Aegerion will need to kick Juxtapid sales growth into high gear just to meet its new, lowered guidance. Where that growth will come from wasn't made clear during Aegerion's conference call Tuesday night.

Juxtapid treats homozygous familial hypercholesterolemia (HoFH), a rare genetic disease that causes extremely high and potentially fatal levels of cholesterol to build up in patients' blood. A year of Juxtapid therapy costs $295,000.

The company says a U.S. sales force expansion and re-alignment, just completed, will be calling on new doctors to identify additional HoFH patients. But investors are growing increasingly skeptical, raising concerns that there are simply far fewer HoFH patients to treat with Juxtapid than the company promised previously.

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